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Latest News: Research

The Basic Cellular Process Of Endocytosis Is Impaired In SMA Models

13 September 2016 / Posted in: Research

Thanks to two recently published scientific studies, we can now add endocytosis to the ever-growing list of pathways that the SMN protein is involved in.

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Avexis Provides Update on Ongoing AVXS-101 Trial

18 August 2016 / Posted in: Research

Avexis has released new information on their ongoing Phase I clinical trial of their gene therapy drug for SMA, AVXS-101.

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Update on Novartis drug LMI070

18 August 2016 / Posted in: Research

The pharmaceutical company Novartis have recently provided an update on their SMN2 splice-modifying drug LMI070.

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New Clinical Care Research Article: End Of Life Care

12 August 2016 / Posted in: Research

In this article, Dr. Alex Murphy describes the sensitivity of discussions around 'end of life care'. He also introduces some of the studies that have been carried out to examine the effectiveness of palliative care.

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Biogen and Ionis Pharmaceuticals' Statement re Nusinersen Announcement

02 August 2016 / Posted in: Research

Biogen and Ionis Pharmaceuticals have announced that Nusinersen has met the primary endpoint in an interim analysis of the phase 3 ENDEAR Study in infants with Infantile-Onset SMA.

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AVXS-101 Receives “Breakthrough Therapy Designation”

28 July 2016 / Posted in: Research

The US Food and Drug Administration (FDA) has granted “Breakthrough Therapy” status to AVXS-101, a gene therapy drug with future potential to treat SMA.

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AAV Therapies report in MDUK’s latest edition of ‘Target Research’

07 July 2016 / Posted in: Research

Dr Haiyan Zhou discusses how gene therapy using harmless viruses called AAVs has great potential as a therapeutic approach to replace defective genes that are responsible for conditions such as SMA and Duchenne muscular dystrophy.

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Can you help identify what questions scoliosis research needs to answer?

30 June 2016 / Posted in: Research

The Scoliosis Research Priority Setting Partnership wants to hear the views of people of all ages with scoliosis, their family, friends and health professionals.

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UK and US Research Interview Study

20 June 2016 / Posted in: Research

The study is looking for participants to help increase understanding of the lived experience of SMA. It aims to support pharmaceutical companies with their research relating to potential treatments in SMA and increase the knowledge of SMA amongst the wider scientific community.

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Novartis Drug Safety Concerns Reported

02 June 2016 / Posted in: Research

Safety concerns have prompted the pharmaceutical company Novartis to pause enrolment in the Phase 2 clinical trial of their SMN2 splice-modifying drug LMI070.

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